CER-001 Therapy as a Novel Approach to Treat Genetic Orphan Diseases

Familial Hypoalphalipoproteinemia Clinical Trial

— TANGO  

Official title:

Phase 3, Multicenter, Randomized, 48 Week, Double Blind, Parallel Group, Placebo Controlled Study to Evaluate Efficacy and Safety of CER-001 on Vessel Wall Area in Patients With Genetically Defined Familial Primary Hypoalphalipoproteinemia

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02697136
• Other study ID #: CER-001-CLIN-009
• Status: Terminated
• Phase: Phase 3
• Start date: December 2015
• Est. completion date: December 21, 2018

Study information

• Verified date: February 2019
• Source: Cerenis Therapeutics, SA
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to assess the impact of 29 intravenous infusions of CER-001 vs. placebo, given at weekly (9 infusions) and biweekly (20 infusions) intervals on carotid vessel wall area as measured by 3TMRI, when administered to patients with familial primary hypoalphalipoproteinemia with proven CVD and appropriate background lipid-lowering therapy.

Description:

Subjects will be required to have acceptable baseline 3TMRI imaging of carotid arteries. Subjects meeting all eligibility criteria will be randomized to receive CER-001 or placebo (2:1 randomization scheme). Randomized subjects will return weekly for a total of 9 infusions and then biweekly for an additional 20 infusions. 3TMRI imaging of the carotid and femoral arteries will be performed at Week 8, Week 24 (primary endpoint) and Week 48. The total study duration from randomization can range from 50 to 54 weeks for patients completing the study as designed.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 30
• Est. completion date: December 21, 2018
• Est. primary completion date: October 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Main Inclusion Criteria:

• Male and female patients, aged 18 and above.

• ApoA-I < 70 mg/dL

• Symptomatic or asymptomatic cardiovascular disease

• Diagnosis of genetically confirmed HDL-c deficiency due to defects in genes coding for ABCA1 and/or ApoA-1

• Stable doses of lipid lowering therapies for at least 6 weeks prior to baseline procedures

Main Exclusion Criteria:

• Females of childbearing potential

• Patients with LCAT mutations

• Patients who experienced recent cardiovascular or cerebrovascular events

• Hypertriglyceridemia (>500 mg/dL)

• Severe anemia (Hgb < 10 g/dL)

• Uncontrolled diabetes (HbA1c >10%)

• Congestive heart failure (NYHA class II or higher)

• Contraindication for MRI scanning (e.g., implanted metal objects, claustrophobia)

Related Conditions & MeSH terms

• Familial Hypoalphalipoproteinemia
• Hypoalphalipoproteinemias

Intervention

Drug:
• CER-001
Recombinant human apoA-I/phospholipid complexes
• Placebo
0.9% Sodium Chloride Injection, USP

Locations

Country	Name	City	State
Belgium	Investigative Site	La Louvière
Canada	Investigative Site	Chicoutimi	Quebec
Canada	Investigative Site	Halifax	Nova Scotia
Canada	Investigative Site	London	Ontario
Canada	Investigative Site	Montreal	Quebec
Canada	Investigative Site	Vancouver	British Columbia
France	Investigative Site	Lille
France	Investigative Site	Montpellier
France	Investigative Site	Rouen
France	Investigative Site	Toulouse
Israel	Investigative Site	Jerusalem
Israel	Investigative Site	Tel Aviv
Italy	Investigative Site	Genoa
Italy	Investigative Site	Milan
Italy	Investigative Site	Pisa
Italy	Investigative Site	Rome
Netherlands	Investigative Site	Amsterdam
Netherlands	Investigative Site	Utrecht
United States	Investigative Site	Salt Lake City	Utah

Sponsors (1)

Lead Sponsor	Collaborator
Cerenis Therapeutics, SA

Countries where clinical trial is conducted

United States,  Belgium,  Canada,  France,  Israel,  Italy,  Netherlands, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Change in femoral MVWA	Assessed by 3TMRI; change from baseline; CER-001 versus placebo	Baseline, Weeks 8, 24 and 48
Primary	Change in mmean vessel wall area (MVWA) of the carotid artery	Change from baseline to Week 24 carotid MVWA; CER-001 versus placebo; measured by 3TMRI	Baseline to Week 24
Secondary	Change in mean vessel wall area (MVWA) of the carotid artery	Change from baseline to Week 8 carotid MVWA; CER-001 versus placebo; measured by 3TMRI	Baseline to Week 8
Secondary	Change in mean vessel wall area (MVWA) of the carotid artery	Change from baseline to Week 48 carotid MVWA; CER-001 versus placebo; measured by 3TMRI	Baseline to Week 48
Secondary	Change in Target to Background Ratio (TBR) of the carotid artery	Change from baseline to Week 24 in carotid TBR; CER-001 versus placebo; measured by FDG-PET	Baseline to Week 24